Wednesday, 22nd June, 2022

14:00 Registration    
16:00 Opening    
16:30 Keynote Lecture Advance in Fukuyama muscular dystrophy & dystroglycanopathy Tatsushi Toda
17:15 Keynote Lecture Exon skipping therapy to Duchenne muscular dystrophy Shin’ichi Takeda
18:00 Peter Harper Memorial Lecture Peter Harper, Myotonic Dystrophy and Me David Brook,
Mark Rogers,
Margaret Bowler
19:00- 20:00 Organization Updates     
Report on International DM Awareness Day    

Thursday, 23rd June, 2022

8:30 Keynote Lecture Lesson from other expansion diseases, disease-modifying therapy for SBMA Gen Sobue

Session 1: Repeat-Associated Pathomechanisms

1-1 Repeat instability
9:15 Overview: Repeat instability Christopher Pearson
9:45 S1-1-1 Identification of CTG repeat contraction factors in myotonic dystrophy type 1  Laure de Pontual
10:00 S1-1-2 CpG sites surrounding DMPK expansions are heterogeneously methylated in myotonic dystrophy type 1 patients with variant repeats Jovan Pesovic
1-2 RNA-mediated mechanism
10:15 Overview: RNA-mediated mechanisms Repeat-mediated pathomechanisms Maurice Swanson
11:00: Break
11:15 S1-2-1 RNA subcellular mislocalization in DM1 patient iPSC-derived neurons Maya L Gosztyla
11:30 S1-2-2 Compensatory mechanism of MBNL paralogs Larissa Nitschke
11:45 S1-2-3 miR-1 and its target Multiplexin are involved in DM1-associated dilated cardiomyopathy  Anissa Souidi
12:00 S1-2-4 Analysis of DMPK expansion transcript degradation and MBNL-RNA binding kinetics in DM1 Xiaomeng Xing
12:15 Lunch
13:15 S1-2-5 Congenital myotonic dystrophy patients exhibit unique patterns of transcriptomic dysregulation independent of CTG repeat expansion  Melissa Ann Hale
13:30 S1-2-6 Multivalency of MBNL is essential for CUG foci formation in DM1 Chase P Kelley
13:45 S1-2-7 Identification of NIPP1 as a modifier of RNA foci formation Yoshihiro Kino
1-3 Cell/organoids and animal models
14:00 S1-3-1 Expanded CUG repeat RNA induces premature senescence in myotonic dystrophy Yuhei Hasuike
14:15 S1-3-2 Manipulating expanded DMPK expression using CRISPRi/a Lise Ripken
14:30 S1-3-3 Generation and Characterization of a DM2 BAC Mouse Model Avery Christian Engelbrecht
14:45 Break
1-4 Tissue specific mechanisms
15:00 S1-4-1 MBNL loss of function in the motor unit alters neuromuscular communication  Charles Frison-Roche
15:15 S1-4-2 Choroid Plexus Spliceopathy in DM1 Benjamin Martin Kidd
15:30 S1-4-3 Cell type-specific abnormalities of central nervous system in myotonic dystrophy type 1 Masayuki Nakamori
15:45 S1-4-4 Sense and antisense RAN proteins accumulate in DM1 brain regions with pathological changes Monica Banez-Coronel
16:00 Mini Lecture  Small Molecules targeting Repeat Sequences causing Neurological Disorders Kazuhiko Nakatani
16:30 Break

Session 2: Clinical Aspects

2-1 Specific disease features
16:45 S2-1-1 An International Consensus Document on Evaluation and Management of Arrhythmias in Myotonic Dystrophy William J. Groh
17:00 S2-1-2 A decade follow-up study of Premanifest DM1: a molecular, muscular and CNS approach Joana Garmendia
17:15 S2-1-3 An integrative analysis of DNA methylation pattern in Myotonic Dystrophy Type 1 samples reveals a distinct DNA methylation profile between tissues and a dual muscle-associated epigenetic dysregulation Monica Suelves
17:30 S2-1-4 Independence of Adults with Childhood Phenotype of Myotonic Dystrophy Type 1 Samar Muslemani
17:45 S2-1-5 TREAT-NMD Myotonic Dystrophy Global Registry Network: An International Collaboration in Myotonic Dystrophy Type 2 Stojan Peric
18:00 Break
2-2 Biomarkers, outcome measures, trial design, etc
18:15 S2-2-1 Longitudinal changes in neuropsychological functioning in Japanese patients with myotonic dystrophy type 1: A 5-year follow-up study Haruo Fujino
18:30 S2-2-2 Blood based biomarker discovery in DM1 Daniel van As
18:45 S2-2-3 miR-223-3p and miR-24-3p as novel serum-based biomarkers for Myotonic Dystrophy type 1 Leonidas A. Phylactou
19:00 S2-2-4 Myotonic dystrophy type I Tau PET imaging and exploratory study of CSF and plasma biomarkers of neurodegeneration from neurocognitively characterized DM1 patients Nicolas Sergeant
19:15 S2-2-5 Analysis of circulating myomiRs as potential biomarkers of progression of muscular impairment in myotonic dystrophy type 1 patients  Nemanja Radovanovic
19:30- 20:00 Overview: Clinical aspect Baziel van Engelen

Friday, 24th June, 2022

Session 3: Therapeutic Strategies and Targets

8:30 S3-01 Whole transcriptome analysis and functional studies reveal that senescence plays a role in Myotonic Dystrophy type 1   Ander Matheu
8:45 S3-02 Correction of Clcn1 mis-splicing reverses muscle fiber type transition in mice with myotonic dystrophy Ningyan Hu
9:00 S3-03 The ReCognitION project: Recognition and Validation of Druggable Targets from the Response to Cognitive Behavioural Therapy in Myotonic Dystrophy type 1 patients from Integrated -Omics Networks Peter-Bram ‘t Hoen
9:15 S3-04 Identifying potential lead molecules that eliminate toxic nuclear foci in DM1 Anjani Kumari
9:30 S3-05 A  CTG  repeat-selective  screen  of  a  natural  product  library reveals dietary natural compounds as potential therapeutics for Myotonic Dystrophy.   Subodh K Mishra
9:45 S3-06 Repeat dosing with DYNE-101 is well tolerated and leads to a sustained reduction of DMPK RNA expression in key muscles for DM1 pathology in hTfR1/DMSXL mice and NHPs  Stefano Zanotti
10:00 Break
10:15 S3-07 Elimination of defective muscle stem cells to restore myogenesis in Myotonic Dystrophy type 1. Nicolas Dumont
10:30 S3-08 Comprehensive transcriptomic characterization of antisense RNA treatments effects on Myotonic dystrophy type 1 cell models Jorge Patricio Espinosa
10:45 S3-09 EEV-Conjugated Oligonucleotide Results in Nuclear Foci Reduction and Aberrant Splicing Correction in DM1 Cell and Animal Models Mahasweta Girgenrath
11:00 S3-10 A Phase 1/2 Trial Evaluating the Safety and Pharmacokinetics (PK) of AOC 1001 in Adults with DM1: MARINA Study Design Nicholas Johnson
11:15 S3-11 Decoy gene therapy to reverse RNA toxicity in DM1  Denis Furling
11:30- 12:00 Closing    

Saturday, June 25, 2022

9:00- 12:30 IDMC-13 Families Day Webinar “Clinical trials have already begun”

AMO Pharma AMO-02 for congenital myotonic dystrophy: an update on progress Mike Snape
Avidity Biosciences Avidity: Ongoing Clinical Trial for DM1 Lisa Ackermann, PhD, SVP Clinical Development.
Dyne Therapeutics Advancing The Treatment Paradigm for Myotonic Dystrophy Type 1 Molly White, Vice President, Global Head of Patient Advocacy and Engagement
Department of Neurology, Osaka University Graduate School of Medicine An investigator-initiated clinical trial (MYD-0124) for treatment of myotonic dystrophy in Japan Masayuki Nakamori, MD., Ph. D.
National Hospital Organization Niigata National Hospital, National Hospital Organization Sendai-Nishitaga Hospital Innovative motor learning therapy with the wearable cyborg Hybrid Assistive Limb, HAL for neuromuscular diseases, including myotonic dystrophy Takashi Nakajima, MD., Ph. D. and Toshiaki Takahashi, MD., Ph. D.
Houston Methodist Neuroscience Research Program Clinical Trials in Myotonic Dystrophy and Beyond Tetsuo Ashizawa, M.D.